Leiden University: Advocating for gene therapies for rare diseases

‘If we don’t start talking about this, who else will?’ This is what Arjan Lankester, paediatrician/immunologist at the Leiden University Medical Center (LUMC) and Professor of Paediatrics, thought when it seemed that various extremely effective gene therapies for rare diseases would no longer be available to patients. Time for action.

So he discussed the matter in a meeting with his English colleague Claire Booth, Professor of Gene Therapy and Paediatric Immunology at UCL Great Ormond Street Institute of Child Health in London. Both knew they could not and would not let this happen. These therapies cure patients. This is how the idea arose of a new European academic group, the AGORA (Access to Gene therapies fOr RAre disease) consortium. Lankester and Booth set to work and in no time had garnered the enthusiasm of a large group of people. The first AGORA meeting was held in London a few weeks ago. ‘It was a really useful first meeting with lots of positive-minded people from all corners of Europe,’ says Lankester.

Quick follow-up to meeting
According to Lankester, there will be a quick follow-up to the meeting. ‘Innovative treatment of rare diseases, often developed with public money, requires a customised approach to secure sustainable access. We hope to enable this process through this consortium.’ The consortium has members from not only different European countries but also different fields, such as academic developers and practitioners, patient groups, funding organisations, regulatory organisations and pharmaceutical companies. And they are all pursuing the goal of creating a sustainable and affordable solution so that patients can continue to access these gene therapies.

Therapies available to patients
The meeting in London was the first brainstorming session and there proved to be great enthusiasm for the topic. Those present recognised how most promising gene therapies for rare diseases that researchers work on don’t reach the patient and agreed that solutions need to be found to this problem. For Lankester and Booth the challenge now is to find the best way to pursue this further. ‘We are going to create a structure to coordinate, support and advise and thus ensure that therapies become and remain available,’ says Lankester.

International response to rare diseases
‘It’ll be a challenge because each country looks at and deals with this in its own particular way, so it’s different in each country. However, everyone agrees that we have to tackle this together. This is also to ensure we don’t all come up with individual solutions to this international problem. You have to tackle rare diseases internationally because of their volume. Then the AGORA consortium would be a connecting and guiding force that ensures everyone is on the same page and that ultimately therapies are not lost or left on the shelf,’ says Lankester.

AGORA to ensure access
Lankester and Booth are currently working on the action items from the meeting. They aim to create a small organisation and are currently exploring – in close consultation with the various stakeholders – how best to do so. The organisation will ensure that therapies that have been developed and cannot be offered in the traditional way are made available at a fair price to patients within this European network. It will also make sure that the proper steps are taken and everything is documented. Lankester and his colleagues aim to have the basic structure in place for AGORA in the new year.